New therapies for tuber-less sclerosis: White matter matters?

摘要

Tuberous sclerosis complex (TSC) is well known to neurologists because of the high prevalence of epilepsy, autism spectrum disorders, and intellectual disabilities seen in individuals with this genetic disease. TSC has been documented in the medical literature for more than 100 years, with clear descriptions of cortical malformations (tubers), skin lesions, epilepsy, and developmental delay.